Outcomes of long-term von Willebrand factor prophylaxis use in von Willebrand disease: A systematic literature review.

Chronic Disease Epistaxis / prevention & control Hospitalization Humans von Willebrand Diseases / complications von Willebrand Factor / therapeutic use
Hemophilia Von Willebrand Disease bleeding disorder bleeding episodes epistaxis prophylaxis

Journal

Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916

Informations de publication

Date de publication:
May 2022
Historique:
revised: 10 01 2022
received: 26 10 2021
accepted: 03 03 2022
pubmed: 27 3 2022
medline: 14 5 2022
entrez: 26 3 2022
Statut: ppublish

Résumé

Von Willebrand Disease (VWD) is a common inherited bleeding disorder. Patients with VWD suffering from severe bleeding may benefit from the use of secondary long-term prophylaxis. Systematically summarize the evidence on the clinical outcomes of secondary long-term prophylaxis in patients with VWD and severe recurrent bleedings. We searched Medline and EMBASE through October 2019 for relevant randomized clinical trials (RCTs) and comparative observational studies (OS) assessing the effects of secondary long-term prophylaxis in patients with VWD. We used Cochrane Risk of Bias (RoB) tool and the RoB for Non-Randomized Studies of interventions (ROBINS-I) tool to assess the quality of the included studies. We conducted random-effects meta-analyses and assessed the certainty of the evidence using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. We included 12 studies. Evidence from one placebo controlled RCT suggested that VWD prophylaxis as compared to no prophylaxis reduced the rate of bleeding episodes (Rate ratio [RR], .24; 95% confidence interval [CI], .17-.35; low certainty evidence), and of epistaxis (RR, .38; 95%CI, .21-.67; moderate certainty evidence), and may increase serious adverse events RR 2.73 (95%CI .12-59.57; low certainty). Evidence from four before-and-after studies in which researchers reported comparative data suggested that VWD prophylaxis reduced the rate of bleeding (RR .34; 95%CI, .25-.46; very low certainty evidence). VWD prophylaxis treatment seems to reduce the risk of spontaneous bleeding, epistaxis, and hospitalizations. More RCTs should be conducted to increase the certainty in these benefits.

Sections du résumé

BACKGROUND BACKGROUND
Von Willebrand Disease (VWD) is a common inherited bleeding disorder. Patients with VWD suffering from severe bleeding may benefit from the use of secondary long-term prophylaxis.
AIM OBJECTIVE
Systematically summarize the evidence on the clinical outcomes of secondary long-term prophylaxis in patients with VWD and severe recurrent bleedings.
METHODS METHODS
We searched Medline and EMBASE through October 2019 for relevant randomized clinical trials (RCTs) and comparative observational studies (OS) assessing the effects of secondary long-term prophylaxis in patients with VWD. We used Cochrane Risk of Bias (RoB) tool and the RoB for Non-Randomized Studies of interventions (ROBINS-I) tool to assess the quality of the included studies. We conducted random-effects meta-analyses and assessed the certainty of the evidence using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach.
RESULTS RESULTS
We included 12 studies. Evidence from one placebo controlled RCT suggested that VWD prophylaxis as compared to no prophylaxis reduced the rate of bleeding episodes (Rate ratio [RR], .24; 95% confidence interval [CI], .17-.35; low certainty evidence), and of epistaxis (RR, .38; 95%CI, .21-.67; moderate certainty evidence), and may increase serious adverse events RR 2.73 (95%CI .12-59.57; low certainty). Evidence from four before-and-after studies in which researchers reported comparative data suggested that VWD prophylaxis reduced the rate of bleeding (RR .34; 95%CI, .25-.46; very low certainty evidence).
CONCLUSION CONCLUSIONS
VWD prophylaxis treatment seems to reduce the risk of spontaneous bleeding, epistaxis, and hospitalizations. More RCTs should be conducted to increase the certainty in these benefits.

Identifiants

DOI: 10.1111/hae.14550 PMID: 35339117
pubmed: 35339117
doi: 10.1111/hae.14550
doi:

Substances chimiques

von Willebrand Factor 0

Types de publication

Journal Article Review Systematic Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

373-387

Subventions

Organisme : American Society of Hematology
Organisme : This systematic review was conducted to support the development of the ASH ISTH NHF WFH 2020 Guidelines for Management of VWD. The entire guideline development process was funded by ASH, ISTH, NHF, and WFH (the 4 collaborating organizations). A.E, N.H., M.K. R.M., R.B.P., and R.A.M. received salary or grant support from the Outcomes and Implementation Research Unit at the University of Kansas Medical Center and the McMaster GRADE center; others participated to fulfill the requirements of an academic degree or program or volunteered their time.

Informations de copyright

© 2022 John Wiley & Sons Ltd.

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Auteurs

Abdallah El Alayli (A)

Outcomes and Implementation Research Unit, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA.
ORCID: 0000-0003-0211-715X

Romina Brignardello Petersen (R)

Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.

Nedaa M Husainat (NM)

Department of Internal Medicine, St. Mary's Hospital, Saint Louis, Missouri, USA.

Mohamad A Kalot (MA)

The State University of New York at Buffalo Department of Internal Medicine, Buffalo, New York, USA.

Yazan Aljabiri (Y)

Lincoln Medical and Mental Health Center, The Bronx, New York, USA.

Hani Turkmani (H)

Department of Cardiovascular Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.

Alec Britt (A)

Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA.

Hussein El-Khechen (H)

Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.

Shaneela Shahid (S)

Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.
Department of Pediatrics, McMaster University, Hamilton, Ontario, Canada.

John Roller (J)

Department of Hematology/Oncology, University of Kansas Medical Center, Kansas City, Kansas, USA.

Shahrzad Motaghi (S)

Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.

Razan Mansour (R)

Outcomes and Implementation Research Unit, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA.

Alberto Tosetto (A)

Hemophilia and Thrombosis Center, Hematology Department, S. Bortolo Hospital, Vicenza, Italy.

Rezan Abdul-Kadir (R)

The Royal Free NHS Foundation Hospital and Institute for Women's Health, University College London, London, UK.
ORCID: 0000-0002-2684-1006

Michael Laffan (M)

Centre for Haematology, Imperial College London, London, UK.
ORCID: 0000-0002-8268-3268

Angela Weyand (A)

Division of Pediatric Hematology Oncology, Department of Pediatrics, University of Michigan Medical School, Ann Arbor, Michigan, USA.
ORCID: 0000-0003-2595-8541

Frank W G Leebeek (FWG)

Department of Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
ORCID: 0000-0001-5677-1371

Alice Arapshian (A)

Middle Village, New York, USA.

Peter Kouides (P)

Mary M. Gooley Hemophilia Treatment Center, University of Rochester, Rochester, New York, USA.

Paula James (P)

Department of Medicine, Queen's University, Kingston, Ontario, Canada.

Nathan T Connell (NT)

Hematology Division, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
ORCID: 0000-0003-4100-7826

Veronica H Flood (VH)

Department of Pediatrics, Medical College of Wisconsin, Versiti Blood Research Institute, Milwaukee, Wisconsin, USA.

Reem A Mustafa (RA)

Division of Nephrology and Hypertension, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA.

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Classifications MeSH

questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Caractéristiques de la maladie › Maladie chronique › Outcomes of long-term von Willebrand factor...
questionsmedicales.fr › États, signes et symptômes pathologiques › Signes et symptômes › Signes et symptômes respiratoires › Épistaxis › Outcomes of long-term von Willebrand factor...
questionsmedicales.fr › Établissements, main d'oeuvre et services de soins de santé › Services de santé › Soins aux patients › Hospitalisation › Outcomes of long-term von Willebrand factor...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Outcomes of long-term von Willebrand factor...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Troubles héréditaires de la coagulation sanguine › Maladies de von Willebrand › Outcomes of long-term von Willebrand factor...
questionsmedicales.fr › Facteurs biologiques › Facteurs de la coagulation sanguine › Facteur de von Willebrand › Outcomes of long-term von Willebrand factor...